Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 20.1 Level: LLT Classification code 10041515 Term: Sphingomyelin lipidosis System Organ Class: 100000004850
Conditions
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The patient and/or patient’s parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed. The patient is aged 0 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The patient has received an investigational drug within 30 days before study enrollment The patient has any of the following medical conditions: - An active, serious, intercurrent illness. - Active hepatitis B or hepatitis C infection. - Infection with human immunodeficiency virus (HIV). - Cirrhosis (determined by clinical evaluation). - Significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or 12 hours a day. The patient, in the investigator’s opinion, is unable to adhere to the requirements of the study. The patient has a platelet count 250 IU/L or total bilirubin >1.5 mg/dL. The patient has an international normalized ratio (INR) >1.5 The patient is unwilling or unable to abstain from ingesting alcohol the day before through 3 days after each infusion of olipudase alfa during the treatment period. Measuring alcohol concentration in blood is not required. The patient is scheduled during the study for in-patient hospitalization including elective surgery. The patient requires medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, or desipramine]). The patient is breast-feeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): Number of adverse events Clinically significant changes in laboratory parameters (complete blood count (CBC), clinical chemistry, and urinalysis) Clinically significant changes in physical examinations (vital signs, electrocardiogram (ECG), doppler echocardiography, and liver ultrasound doppler);Timepoint(s) of evaluation of this end point: From screening through week 64;Main Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 64 weeks;Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 64 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Maximum concentration (Cmax) Area under the curve until the last measurable concentration (AUClast) Area under the curve extrapolated to infinity (AUC) Half-life (t1/2) Clearance (CL) Volume of distribution (Vss) Change in sphingomyelin levels Change in sphingomyelin metabolite levels;Timepoint(s) of evaluation of this end point: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at week 52 Maximum concentration (Cmax) Area under the curve until the last measurable concentration (AUClast) Area under the curve extrapolated to infinity (AUC) Half-life (t1/2) Clearance (CL) Volume of distribution (Vss) From Day 1 through Week 64 Change in sphingomyelin levels Change in sphingomyelin metabolite levels | — |
Countries
Brazil, France, Germany, Italy, United Kingdom, United States
Contacts
Public ContactMedical Information
Genzyme Europe B.V.
Outcome results
None listed